Bayer AG and its subsidiary BlueRock Therapeutics announced positive 24-month data from the Phase 1 exPDite clinical trial of bemdaneprocel, an investigational cell therapy for Parkinson's disease. The data, presented at the International Congress of Parkinson's Disease and Movement Disorders, indicate a favorable safety profile and encouraging trends in motor symptom improvement.
Favorable Safety Profile and Cell Survival
The exPDite trial, designed to assess the safety and tolerability of bemdaneprocel, showed that the therapy continues to be well-tolerated by patients, with no adverse events reported related to the treatment. Notably, transplanted cells continued to survive and engraft in the brain even after the discontinuation of immunosuppression therapy at 12 months, as per the study protocol.
Motor Symptom Improvement
Secondary clinical endpoints related to motor symptoms demonstrated positive trends from baseline through the 24-month follow-up. The high-dose cohort (2.7 million cells per putamen) showed more encouraging trends than the low-dose cohort (0.9 million cells per putamen). These improvements were assessed using the MDS-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and the Hauser PD Diary.
In the high-dose cohort, the 24-month measurement of motor symptoms using MDS-UPDRS Part III in the “OFF”-medication state showed a mean reduction of 21.9 points compared to baseline. The low-dose cohort showed a mean decrease of 8.3 points. Using the Hauser PD Diary, participants in the high dose cohort showed a mean increase of 1.8 hours in time spent in the “Good ON” state without troublesome dyskinesias compared with baseline after 24 months. Time spent in the “OFF” state showed a mean decrease of 1.9 hours from baseline after 24 months.
Expert Commentary
"There is considerable momentum in the concept of restoring dopamine inputs in the brain using transplanted cells, and the positive results from the exPDite trial leads the drive forward," said Claire Henchcliffe, MD, chair of the UCI School of Medicine Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators. "The completed study demonstrates that the transplanted cells survive and there are early signs that bemdaneprocel can potentially help patients to better control their motor symptoms. These are exciting results that warrant further exploration in a next phase placebo-controlled study."
Bemdaneprocel and the exPDite Trial
Bemdaneprocel is an investigational cell therapy designed to replace dopamine-producing neurons lost in Parkinson's disease. The therapy involves the transplantation of dopaminergic neuron precursors, derived from human embryonic stem cells, into the brain. The exPDite trial is a Phase 1, multi-center, open-label, non-randomized study involving 12 participants diagnosed with Parkinson’s disease. The primary endpoint was safety and tolerability at 12 months, with secondary endpoints assessing cell survival and motor effects.
Regulatory Status and Future Directions
In May 2024, bemdaneprocel received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, which may expedite its development and review. Further placebo-controlled studies are planned to explore the therapy's potential benefits further.
"The continued positive results of the exploratory clinical endpoints for bemdaneprocel after 24 months are encouraging and support our commitment in developing innovative therapies that can significantly improve patient lives," said Christian Rommel, Head of Research and Development at Bayer’s Pharmaceuticals Division.
